Congressional Bill Supporting the Continued Development of Medically Formulated GHB Moves to the President

See Also: CCLE GHB Index

Updated 4:51 PM ET February 1, 2000

MINNEAPOLIS, Feb. 1 /PRNewswire/ --

The U.S. House of Representatives approved by a vote of 339-2 federal legislation making gamma hydroxybutyrate (GHB) illegal while allowing medical formulations to be available for clinical testing and potential medical use.

The bill was sent to the President and will become law with his signature. Orphan Medical, Inc. is evaluating Xyrem(R) (sodium oxybate) oral solution, a medically formulated GHB, in the treatment of narcolepsy, a chronic and debilitating sleep disorder.

The bill directs the U.S. Attorney General to use her emergency scheduling authority to make illicitly made GHB a Schedule I substance within 60 days. Schedule I is the designation by which illegal drugs such as heroin are controlled.

The bill also says the Attorney General's order must treat medically-formulated GHB products, such as Orphan Medical's Xyrem, as a Schedule III substance which will allow for continued research and development.

Orphan Medical recently reported results of clinical trials in which Xyrem was assessed as a treatment for narcolepsy, and particularly the symptom of cataplexy. Cataplexy is a loss of muscle control that occurs in response to strong emotional reactions such as laughter, anger, or surprise and can cause a person to fall or collapse during waking hours. The condition occurs in up to 60-75% of an estimated 125,000 patients with narcolepsy, and is currently treated with anti-depressants, selective serotonin uptake inhibitors, and tricyclic antidepressants. Long-term treatment with these REM suppressing compounds may be limited by efficacy, tolerance and adverse side effects such as dry mouth, weight gain, tachycardia and loss of libido.

John Bullion, CEO of Orphan Medical said, "We believe that Orphan Medical's recently reported Xyrem clinical trials results strongly indicate that Xyrem may be an important treatment for narcolepsy. Cataplexy is poorly treated by current therapies and Xyrem represents a potential effective long- term treatment for this chronic disease. We plan to submit an NDA for Xyrem to the FDA later this year, and expect that it will be granted a priority review. If the FDA approves Xyrem, it will be an important new drug for the treatment of narcolepsy. This bill gives law enforcement officials the tools they need to punish those who use GHB illicitly and to commit crimes, while protecting patient access to a promising new drug."

Abbey Meyers, President of the National Organization of Rare Disorders, said, "This legislation recognizes the critical need of patients with this rare disorder and does not preclude the scientific and clinical assessment of GHB for medical therapy. Much more attention needs to be directed toward the development of drugs for rare disorders. This bill recognizes the valid medical use of a drug for a seriously ill population of patients that have been neglected by medicine until now."

Bob Cloud, President of Narcolepsy Network, commented, "This appears to be an important treatment for narcolepsy. I'm aware of many patients enrolled in Orphan Medical's clinical trials who, prior to receiving Xyrem, could not function on a daily basis, and now, with Xyrem, can hold jobs, drive, and perform functions that most of us take for granted. This legislation provides hope to all narcolepsy patients that Xyrem's potential as a treatment for narcolepsy or its symptoms will not be eliminated by irresponsible use of GHB."

Orphan Medical, Inc. is dedicated to patients with inadequately treated and uncommon diseases. To that end, the acquires, develops, and markets products of high medical value for patients within selected strategic therapeutic market segments. Orphan Medical's Internet Web Site address is http://www.orphan.com/

The information in this press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. A number of factors could cause actual results to differ materially from the Company's assumptions and expectations. These are set forth in the cautionary statements included in Exhibit 99 to Orphan Medical's most recent Form 10-Q or Form 10-K filed with the Securities and Exchange Commission. All forward-looking statements are qualified by, and should be considered in conjunction with, such cautionary statements.

Contact: John Howell Bullion of Orphan Medical, Inc., 612-513-6900